Actively Recruiting

Phase Not Applicable
Age: 18Years - 80Years
MALE
NCT04817462

Liver Biopsy In Haemophilia Gene Therapy

Led by University College, London · Updated on 2024-12-20

10

Participants Needed

1

Research Sites

151 weeks

Total Duration

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AI-Summary

What this Trial Is About

To perform a liver biopsy in haemophilia A and B patients with endogenous FVIII:C/FIX:C expression at \>1% any time after gene transfer following AAV mediated gene transfer. This is to obtain tissue for analysis, to understand if FIX/FVIII transgenic protein expression is mediated by AAV proviral DNA that is integrated into the host cell DNA or if stable expression in humans is mediated by episomal maintained AAV genome.

CONDITIONS

Official Title

Liver Biopsy In Haemophilia Gene Therapy

Who Can Participate

Age: 18Years - 80Years
MALE

Eligibility Criteria

Eligible

You may qualify if you...

  • Male aged 18 to 80 years old
  • Previously treated with gene therapy in one of the following trials at Royal Free Hospital: AGT4HB (FIX), GO-8 (FVIII), or FLT180a-01 (FIX)
  • Endogenous factor VIII:C or IX:C expression greater than 1% anytime after gene transfer
  • Normal prothrombin time (PT) and thrombin time (TT) as shown in coagulation tests
Not Eligible

You will not qualify if you...

  • Platelet count less than 140 x10^9/L
  • Any condition that would prevent full compliance with the study or affect safety and evaluation
  • Abnormal kidney function with estimated GFR less than 50 ml/min
  • Known allergy to iodine-based intravenous contrast agents
  • Known allergy to local or general anesthesia
  • Known reaction to factor VIII or IX concentrate infusions
  • Presence of factor VIII or IX inhibitors within 14 weeks before biopsy
  • Any bleeding disorder unrelated to hemophilia A or B
  • Unable or unwilling to provide informed consent

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 1 location

1

Royal Free Hospital

London, United Kingdom, NW3 2QG

Actively Recruiting

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Research Team

P

Paul Batty

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

BASIC_SCIENCE

Number of Arms

1

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